Growing up with Charcot-Marie-Tooth disease (CMT), Lainie Ishbia learned about living with invisible disability — experiencing the daily struggle of movement without looking, outwardly and obviously, as if that’s the case. She also learned, once ankle-foot orthotics (AFOs, or braces) made her disability visible, those devices designed to help make movement easier can also make . . .
Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared . . .
At 22, when I graduated from college, I, like so many of my fellow millennials, moved back home. I spent two transitional years in the room where I’d grown up, unicorn wallpaper still casting magic above the bed, and then, one weekend, I packed my clothes and moved out. I’d lived alone in my college . . .
In honor of Charcot-Marie-Tooth (CMT) Awareness Month, the Muscular Dystrophy Association joins the Charcot-Marie-Tooth Association (CMTA) and the Hereditary Neuropathy Foundation (HNF) to host an externally led patient-focused drug development (PFDD) meeting for CMT. The PFDD meeting is being held on Friday, Sept. 28 at the College Park Marriott Hotel and Conference Center in Hyattsville, . . .
The future of Charcot-Marie-Tooth research and treatments rests where the disease begins: in genes. While many new genes involved in CMT have been identified, we know as many as 40 percent of people living with CMT still don’t have a confirmed genetic diagnosis. We’re also still trying to understand the disease mechanisms of the genes . . .
The Muscular Dystrophy Association recently awarded an MDA human clinical trial grant for the development of a critical biomarker for Charcot-Marie-Tooth disease (CMT) to Mary M. Reilly, professor of clinical neurology and consultant neurologist, UCL Institute of Neurology and National Hospital for Neurology and Neurosurgery, London. The investment, totaling $1 million over three years, will . . .
Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT. FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT. Trial length is approximately three . . .